Abstract: Compounds, and pharmaceutically acceptable salts thereof, useful as inhibitors of sodium channels are provided. Also provided are pharmaceutical compositions comprising the compounds or pharmaceutically acceptable salts and methods of using the compounds, pharmaceutically acceptable salts, and pharmaceutical compositions in the treatment of various disorders, including pain.

Abstract: Provided herein are processes for preparing an oligomer (e.g., a morpholino oligomer). The synthetic processes described herein may be advantageous to scaling up oligomersynthesis while maintaining overall yield and purity of a synthesized oligomer.

Abstract: The present invention discloses a method of treating tumor using a novel pyrimidine derivative. It is demonstrated through the tests on anti-tumor activity at the cellular level (tumor cells expressed with c-Met) that, through the introduction of a chiral structure in the molecule, the introduction of an isotope of hydrogen (deuterium) at the sites that are prone to metabolism in the molecule, and the introduction of atoms or groups such as sulfur, selenium and sulfoxide in the molecule, the compounds of the invention have excellent anti-tumor activities, and the stabilities of the anti-tumor compounds are significantly improved.

Abstract: FGFR3-related chondrodysplasias represent a group of rare diseases. Among them, achondroplasia, a nonlethal form of chondrodysplasia, is the most common type of dwarfism. The mutation, which produce an increase of FGFR3 function, affects many tissues, most strikingly the cartilaginous growth plate and bone in the growing skeleton, leading to a variety of manifestations and complications. In attempt to find a new therapeutic approach for FGFR3-related chondrodysplasia, the inventors purified (?)-epicatechin from T. cacao and showed that (?)-epicatechin treatment significantly increases the length of the Fgfr3Y367C/+ femurs comparing to Fgfr3+/+ femurs and improves the whole growth plate cartilage. The present invention thus relates to the use of (?)-epicatechin for the treatment of FGFR3-related chondrodysplasias.

Abstract: Provided herein are compounds, pharmaceutical compositions, and methods of treatment for various diseases or conditions, such as cancer. In one aspect, the method comprises the treatment of metastatic cancers. Compounds and methods provided herein are also used for the treatment of diseases such as inflammatory disease, cardiovascular disease, autoimmune disease, and dry eye syndrome. Further provided herein are dietary supplement formulations and methods for supporting a healthy lifestyle.

Abstract: Hydrophilic Linkers, Drug-Linker compounds, Drug-Ligand Conjugate compounds and Ligand-Linkers and methods of making and using the same are provided.

Abstract: The invention provides novel sugar alcohol-based dendrimer-like molecules. These sugar alcohol-based molecules have multiple functional groups that can be used to label, conjugate, and immobilize large amounts of molecules or multiple types of molecules of interest for diagnostic, therapeutic, and research usage. Methods of synthesizing these sugar alcohol-based dendrimer-like molecules and conjugates are also within the scope of the invention.

Abstract: The present disclosure relates to compounds of formula (I) and pharmaceutical compositions and their use in reducing Widely Interspaced Zinc Finger Motifs (WIZ) expression levels, or inducing fetal hemoglobin (HbF) expression, and in the treatment of inherited blood disorders (e.g., hemoglobinopathies, e.g., beta-hemoglobinopathies), such as sickle cell disease and beta-thalassemia.

Abstract: The present invention provides a cyclobutyl dihydroquinoline sulfonamide compound of Formula (I), an enantiomer, diastereoisomer, atropisomer thereof, a mixture thereof, or a pharmaceutically acceptable salt thereof, that inhibits voltage-gated sodium channels, in particular Nav1.7. The compounds are useful for the treatment of diseases associated with the activity of sodium channels such as pain disorders, cough, and itch. Also provided are pharmaceutical compositions containing the compounds of the present invention. Also further provided is an atropi-selective preparation of said compounds of Formula (I), and intermediate thereof.

Abstract: An organometallic compound represented by Formula 1: M(L1)n1(L2)n2??Formula 1 In Formula 1, M is a transition metal; L1 is a ligand represented by Formula 2 as disclosed herein; L2 is a monodentate ligand, a bidentate ligand, a tridentate ligand, or a tetradentate ligand; n1 is 1, 2, or 3, wherein, when n1 is 2 or greater, two or more ligands L1 are identical to or different from each other; n2 is 0, 1, 2, 3, or 4, wherein, when n2 is 2 or greater, two or more ligands L2 are identical to or different from each other; and L1 and L2 are different from each other.

Abstract: The present invention provides for the treatment, prevention, and/or reduction of a risk of a disease, disorder, or condition in which aldehyde toxicity is implicated in the pathogenesis, including ocular disorders, skin disorders, conditions associated with injurious effects from blister agents, and autoimmune, inflammatory, neurological and cardiovascular diseases by the use of a primary amine to scavenge toxic aldehydes, such as MDA and HNE.

Abstract: A compound having the following structure: wherein: R1 is a linker having the formula —(CH2)n-L1-, wherein L1 contains 1-6 carbon atoms and at least one —NH— linkage and at least one oxygen-containing or sulfur-containing linkage, and n is an integer of 0-3; R2 is a group having the formula —C(Y)-E, wherein Y is O or S, and E is a hydrocarbon group and either: (i) at least one carbon-carbon or carbon-nitrogen unsaturated bond or (ii) at least one alkyl halide group; R3 is selected from H, NR?2, NHC(O)R?, and —(CH2)p-T, wherein T contains at least one —NH— linkage; Ra, Rb, Rc, Rd, Re, and Rf are independently selected from of H, hydrocarbon groups containing 1-3 carbon atoms, fluorine atom, and chlorine atom; X is N or CR9, wherein R9 is selected from H, hydrocarbon groups containing 1-3 carbon atoms, fluorine atom, and chlorine atom; and pharmaceutically acceptable salts thereof.

Abstract: The invention provides methods of treatment of non-obstructive hypertrophic cardiomyopathy using modified forms of trimetazidine, such as CV-8972 and CV-8814.

Abstract: A scaled process of preparing maytansinol, FORMULA II, is provided by reacting a compound of Formula I, wherein R is selected from the group consisting essentially of alkyl, branched alkyl, aryl, alkenyl, alkynyl, as well as substituted variations thereof, with at least one organometallic reagent to produce the compound of Formula II; and, isolating the compound of Formula II.

Abstract: The present disclosure is concerned with benzimidazole compounds and methods of using these compounds for the treatment of hepatitis (e.g., hepatitis C), RNA virus infections (e.g., Zika virus, dengue virus, Powassan virus, Chikungunya virus, Enterovirus, respiratory syntactical virus (RSV), Rift Valley fever, Influenza virus, Tacaribe virus, Mayaro virus, West Nile virus, yellow fever virus, and coronavirus), and disorders of uncontrolled cellular proliferation (e.g., cancer). This abstract is intended as a scanning tool for purposes of searching in the particular art and is not intended to be limiting of the present invention.

Abstract: A method of inhibiting TNIK and/or MAP4K4 kinase can include: contacting the kinase with a compound of Formula A, wherein: ring 1 is an aromatic ring with or without hetero atoms; ring 2 is a hetero aromatic ring; ring 3 includes at least one hetero aromatic ring and optionally at least one cycloaliphatic ring fused with the at least one hetero aromatic ring; ring 4 is an aromatic ring with or without hetero atoms; Y is a bond or a linker; Y1 is a linker; each n is independently 0, 1, or 2; each o is independently 0. 1, 2, 3, 4, or 5; each R1, R6, R11, and R12 is independently a substituent; and RA is a ring structure, straight aliphatics, or branched aliphatics, which can be substituted or unsubstituted, any with or without hetero atoms.

Abstract: The present invention pertains to a pharmaceutical composition for the prevention or treatment of kidney disease, a kidney inflammation inhibitor, an inflammatory cell death inhibitor for the kidneys, or a food for the prevention or improvement of kidney disease, that include D-alanine or a derivative thereof. In addition, the present invention pertains to the use of D-alanine or a derivative thereof, for the production of a pharmaceutical composition for the prevention or treatment of kidney disease.

Abstract: Scaffold compounds are used to design small molecule compounds and structure-activity relationship studies identify inhibitors of inflammation. Methods include pharmaceutical compositions of the small molecule compounds to inhibit, prevent and treat diseases and conditions associated with inflammation, including multiple sclerosis, Alzheimer's disease, acute myocardial infarction, traumatic brain injury and autoinflammatory diseases.

Abstract: Provided herein are pharmaceutical compositions, each comprising a pyrone compound, for example, a compound of Formula I, and a pharmaceutically acceptable excipient. Also provided herein are methods of their use for treating, preventing, or ameliorating one or more symptoms of an inflammatory, neurodegenerative, or immune-mediated disease (e.g., multiple sclerosis).

Abstract: Disclosed is chemical compounds C that are derivatives of norbixin and have tropism for the eye, and are intended to be used in the treatment of eye diseases in mammals, in particular in the context of altering the retinal pigment epithelium and more particularly in the context of age-related macular degeneration (AMD) and Stargardt's disease.